Press Release

EffRx Pharmaceuticals announces the launch of Alkindi® as replacement therapy for pediatric adrenal insufficiency in Switzerland

July 20, 2022

EffRx Pharmaceuticals announces the launch of Alkindi® as replacement therapy for pediatric adrenal insufficiency in Switzerland

FREIENBACH, Switzerland – EffRx Pharmaceuticals SA, a commercial-stage company that commercializes niche and orphan medicines in Switzerland and Europe, today announces the launch of Alkindi® (hydrocortisone capsules for oral use) in Switzerland as replacement therapy for adrenal insufficiency in infants, children and adolescents (from birth to <18 years old).

Alkindi® is the first approved pediatric-specific hydrocortisone formulation for the replacement therapy for adrenal insufficiency (AI) in pediatric patients. Alkindi® is available in color-coded capsules with four different dose strengths (0.5 mg, 1 mg, 2 mg, 5 mg) and allows for individualized, accurate and flexible dosing – without the need for magisterial formulations or tablet fragmentation.

Pediatric AI, including the related genetic condition congenital adrenal hyperplasia (CAH), is a condition characterized by a deficiency in cortisol. Untreated, the disease is associated with significant morbidity and increased mortality. In Switzerland, there are approximately 200 patients suffering from pediatric AI. This condition in children is currently treated by compounding hydrocortisone or crushing/splitting tablets of adult preparations with the potential risks of lack of efficacy and/or increased side effects.

Dr. Lorraine Zakin, Senior Director Medical Affairs at EffRx commented: “I am very pleased that this medicine, specifically studied in children and designed to treat children with adrenal insufficiency, is available in Switzerland. Alkindi® addresses the shortfall in pediatric-specific medicines.”

Alkindi® advances the therapeutic options to help young patients suffering from pediatric AI and CAH and provides the first regulated, consistent preparation of hydrocortisone to ensure efficacy, safety and ease of use for children suffering from AI and their caregivers.

About Pediatric Adrenal Insufficiency (AI) and congenital adrenal hyperplasia (CAH)

Pediatric AI and the related genetic condition CAH are characterized by deficiency in cortisol, an essential hormone in regulating metabolism and the response to stress. The primary symptoms of AI are chronic fatigue, risk of adrenal crisis and death in case of inadequate cortisol replacement.

About Alkindi®

Alkindi® is the first preparation of hydrocortisone specifically designed for use in children suffering from pediatric AI. Alkindi® is a patented, oral, immediate-release pediatric formulation of hydrocortisone granules in capsules for opening that allows for accurate, yet flexible and personalized dosing in children. This therapeutic approach has the potential to help young patients less than eighteen years of age suffering from pediatric AI and the related condition CAH.

About EffRx Pharmaceuticals

EffRx Pharmaceuticals is a commercial-stage pharmaceutical company focused on the late-stage development and commercialization of medicines for niche and orphan indications. The business model is centered around providing superior clinical value propositions for physicians, payers and patients.

EffRx pro-actively seeks in-licensing opportunities for Switzerland and Europe in niche therapeutic areas, with a primary interest for rare diseases. EffRx’s go-to-market competence is proven by the development, launch and expansion of Binosto® in highly competitive European markets as well as US, selected MEA and Asian countries.